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HOST-VECTOR SYSTEM FOR USE IN GENE THERAPY

机译:用于基因治疗的宿主系统

摘要

The invention relates to a system for expressing a transgene in a target cell or a human or animal tissue characterized in that it consists of a eukaryotic cell and established in line, in which were transfected: a) a recombinant viral sequence in which a gene has been deleted in whole or in part and substituted by said transgene at this gene; b) a nucleic acid sequence including a sequence encoding the deleted protein, which sequence is under the control of a promoter and is associated where appropriate to said transgene, and flanked at its 3 'end a polyadenylation site; said recombinant viral genome and said sequence, carried by one or two plasmid supports, being capable of complementing each other in trans and allowing said host cell to produce defective infectious viruses.
机译:本发明涉及用于在靶细胞或人或动物组织中表达转基因的系统,其特征在于它由真核细胞组成并且被建立并在其中被转染:a)重组病毒序列,其中基因具有全部或部分缺失,并在该基因上被所述转基因取代; b)包含编码缺失蛋白的序列的核酸序列,该序列在启动子的控制下并在适当的情况下与所述转基因相关联,并在其3'端侧翼为聚腺苷酸化位点;由一个或两个质粒支持物携带的所述重组病毒基因组和所述序列能够反式互补,并允许所述宿主细胞产生有缺陷的感染性病毒。

著录项

  • 公开/公告号CA2183151C

    专利类型

  • 公开/公告日2008-12-09

    原文格式PDF

  • 申请/专利权人 UNIVERSITE PIERRE ET MARIE CURIE (PARIS VI);

    申请/专利号CA19952183151

  • 发明设计人 KLATZMANN DAVID;SALZMANN JEAN-LOUP;

    申请日1995-02-22

  • 分类号C12N15/86;A61K48;C12N7;C12N7/04;C12N9/12;C12N15/864;C12N15/867;C12N15/87;

  • 国家 CA

  • 入库时间 2022-08-21 19:23:40

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